SAN RAFAEL, Calif.,
Sept. 14, 2017 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced
that the U.S. Food and Drug Administration (FDA) has provided the
company with the "Day-74" filing communication for its Biologics
License Application (BLA) for pegvaliase, a PEGylated recombinant
phenylalanine ammonia lyase enzyme product, to reduce blood
phenylalanine (Phe) levels in adult patients with phenylketonuria
(PKU) who have uncontrolled blood Phe levels on existing
management. In the letter, the FDA advised the Company that
it is not currently planning to hold an advisory committee meeting
to discuss the application. Additionally, the FDA indicated
that the review is proceeding according to the Agency's internal
review timelines described in their Guidance on Good Review
Management Principles and Practices for PDUFA Products. The
FDA comments based on its preliminary evaluation are generally
in-line with the Company's expectations.
"We are pleased with our ongoing interactions with the FDA on
the pegvaliase BLA. While the Agency has indicated they do
not currently plan to hold an advisory committee meeting, the FDA
may still consult with outside experts and patients in a similar
fashion as they did when reviewing the BLA for our most recently
approved treatment for a form of Batten disease," said Hank Fuchs, M.D., President Worldwide Research
and Development at BioMarin. "We continue to work closely
with the FDA as they review this application, and we are confident
that we will be able to respond to the Agency's comments in a
timely manner."
The FDA has granted priority review designation to pegvaliase,
which is granted to drugs that treat a serious condition and, if
approved, would provide a significant improvement in safety or
effectiveness of the treatment, prevention, or diagnosis of a
serious condition. The PDUFA action date is February 28, 2018. However, the FDA has
requested additional Chemistry, Manufacturing, and Controls (CMC)
information, which we expect, when submitted, will be classified as
a major amendment and result in a three month extension of the
PDUFA date.
About Pegvaliase
Pegvaliase is an investigational study drug that substitutes the
deficient PAH enzyme in PKU with the PEGylated version of the
enzyme phenylalanine ammonia lyase, to break down Phe. It is being
developed as a potential treatment for adults with inadequately
controlled blood Phe levels in the study. In clinical
studies, treatment with subcutaneous pegvaliase substantially
reduced blood Phe compared to placebo using a randomized withdrawal
study design, and led to long-term maintenance of Phe reduction in
the majority of adult patients with PKU. Pegvaliase was
administered using a dosing regimen that achieved a manageable
safety profile, consisting primarily of immune-mediated responses,
including anaphylaxis, for which robust risk management measures
effective in clinical trials will be proposed.
For additional information regarding the investigational product
pegvaliase, please contact BioMarin Medical Information at
medinfo@bmrn.com.
About Phenylketonuria
Phenylketonuria (PKU), or phenylalanine hydroxylase (PAH)
deficiency, is a genetic disorder affecting approximately 50,000
diagnosed patients in the developed world and is caused by a
deficiency of the enzyme PAH. This enzyme is required for the
metabolism of Phe, an essential amino acid found in most
protein-containing foods. If the active enzyme is not present in
sufficient quantities, Phe accumulates to abnormally high levels in
the blood and becomes toxic to the brain, resulting in a variety of
complications including severe intellectual disability, seizures,
tremors, behavioral problems and psychiatric symptoms. As a result
of newborn screening efforts implemented in the 1960s and early
1970s, virtually all individuals with PKU under the age of 40 in
developed countries are diagnosed at birth and treatment is
implemented soon after. PKU can be managed with a Phe-restricted
diet, which is supplemented by low-protein modified foods and
Phe-free medical foods; however, the strict diet is difficult for
most patients to adhere to the extent needed for achieving adequate
control of blood Phe levels.
To learn more about PKU, please visit www.PKU.com. Information
on this website is not incorporated by reference into this press
release.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. The company's portfolio consists
of six commercialized products and multiple clinical and
pre-clinical product candidates.
For additional information, please visit www.BMRN.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: BioMarin's product candidate
pegvaliase, and specifically about expectations regarding the BLA
filing for pegvaliase with the FDA and the FDA's evaluation of such
filing; expectations regarding an advisory committee meeting to
discuss the application; the potential outcome of the review of
such filings; and the possible approval of such product
candidate. These forward-looking statements are predictions
and involve risks and uncertainties such that actual results may
differ materially from these statements. These risks and
uncertainties include, among others: results and timing of current
and planned clinical trials of its product candidates; the content
and timing of decisions by the FDA, the EMA and other regulatory
authorities concerning pegvaliase; and those other risks detailed
from time to time under the caption "Risk Factors" and elsewhere in
the Company's Securities and Exchange Commission (SEC) filings,
including the Company's Quarterly Report on Form 10-Q for the
quarter ended June 30, 2017, and
future filings and reports by the Company. The Company undertakes
no duty or obligation to update any forward-looking statements
contained in this press release as a result of new information,
future events or changes in its expectations.
BioMarin® is a registered trademark of BioMarin Pharmaceutical
Inc.
Contact:
|
Investors:
|
Media:
|
Traci
McCarty
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(415)
455-7451
|
View original content with
multimedia:http://www.prnewswire.com/news-releases/fda-not-currently-planning-to-hold-advisory-committee-meeting-for-biomarins-pegvaliase-biologics-license-application-bla-300520020.html
SOURCE BioMarin Pharmaceutical Inc.